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Measuring the survival of transfused red blood cells in patients with Sickle cell disease and b-Thalassemia: A Biotin Label approach

To assess what the survival of transfused red blood cells (RBC) is in patients with sickle cell disease and *-thalassemia.

Sickle Cell Outcome REsearch

To determine the natural history of sickle cell disease and to identify modifying factors, including: (epi)genetic, biological, pathophysiological, social/ demographic, psychological and therapeutic determinants, which contribute to morbidity and…

Impact of Alemtuzumab exposure on immune reconstitution, autoimmunity, risk of infection, chimerism and Graft-versus-Host Disease in children with non-malignant diseases undergoing allogeneic stem cell transplantation. ARTIC International Multicenter Observational Study.

Primary objectiveTo evaluate current clinical practice and develop a population based Campath® (Alemtuzumab) pharmacokinetics model for predicting total Alemtuzumab exposure after i.v. administration before allogeneic stem cell transplantation in…

A Phase II, multicenter, randomized, open label two arm study comparing the effect of crizanlizumab + standard of care to standard of care alone on renal function in sickle cell disease patients * 16 years with chronic kidney disease due to sickle cell nephropathy

To evaluate the effect of crizanlizumab + standard of care compared to standard of care alone on albuminuria (ACR) decrease at 12 months